Cost-effectiveness of pasireotide as second-line therapy for adult acromegaly patients from the perspective of the Brazilian Unified Health System.

BACKGROUND

Acromegaly is a rare, chronic disease caused by excessive growth hormone (GH) production due to pituitary adenoma. The disease severely impacts patients' health-related quality of life and mortality while imposing a substantial economic burden. Resistance to first-line medical treatment necessitates effective second-line therapies, including pasireotide long-acting release (PAS LAR) and pegvisomant (PEG).

OBJECTIVE

To evaluate the cost-effectiveness of PAS LAR versus PEG (monotherapy or combined with FGSRLs) and versus FGSRLs alone, as second-line medical treatments for adults with acromegaly who are not adequately controlled on FGSRLs, from the perspective of the Brazilian Unified Health System (SUS).

METHODS

A hybrid decision-tree and Markov model were developed to evaluate lifetime health and economic outcomes of assessed drugs. To inform the model, health outcomes data were sourced from clinical trials, real-world evidence, systematic reviews, and meta-analyses. Costs were extracted from the official Brazilian tariffs database. Comparators included the FGSRLs, lanreotide (LAN) and octreotide (OCT), and PEG, as monotherapy and in combination with FGSRLs. Deterministic and probabilistic sensitivity analysis were performed to assess the impact of uncertainty on results. The incremental cost-effectiveness ratio (ICER) was the primary analysis outcome.

RESULTS

PAS LAR was dominant versus PEG monotherapy and in combination with LAN, generating similar health benefits (an additional 0.17 and 0.10 QALYs, respectively) and lower therapy costs (savings of -R$538,945 [-US$100,675] and -R$782,542 [-US$146,179]). In the comparison with PEG combined with OCT, PAS LAR was less costly (-R$979,624 [-US$182,994]) and marginally less effective (-0.04 QALYs). Compared to FGSRLs, PAS LAR demonstrated improved health outcomes (QALYs) and increased cost. The ICERs were R$161,411 (US$30,152)/QALY and R$168,538 (US$31,483)/QALY versus OCT and LAN, respectively. Scenario analyses confirmed robustness across variations in key model inputs that have the highest impact on cost-effectiveness results.

CONCLUSIONS

The findings showed that in the Brazilian SUS context, PAS LAR is cost-effective compared to PEG monotherapy and PEG + LAN, and cost-saving versus PEG + OCT. Compared to FGSRLs, PAS LAR provided better outcomes with higher costs. With limited options for second-line medical treatment in Brazil, the reimbursement of PAS LAR in acromegaly can be considered a cost-effective strategy for acromegaly patients.